Controlling Trials

You quote: It must be difficult to justify giving one set of patients the proposed (nutriceutical) drug and another just the placebo when you know they are receiving nothing and the only benefit they will get is the placebo effect, whilst there are DMT's that could help them already on the market.


Remember previous posts such as Do you think placebo-controlled trials are ethical?


Has G changed his mind One Year On?-Probably not, because the promise of effective first-line oral agents has sadly still yet to materialise within the UK, but this is changing elsewhere.


In RR MSers there has been an increasing number of clinical trials comparing active drug typically compared with an injectable such as interferon rather than placebo. This speaks to avoiding an ethical issue of MSer's not gettting no active treatment.



However, it also has to be said that this provides a real marketing oppertunity to show that the new test drug is better than current, competitor drugs. Examples of this could be the efficacy of alemtuzumab over interferon beta. These lack a placebo arm, but are not really double-blinded (MSersand and treating doctor don't know treatment drug) when comparing daily injectables with intravenous injectables. Should we make antibodies infused monthly to go aginst tysabri, which is highly effecicacious? This unlikely to occur as it has the risk to backfire when the test drug has worse efficacy than the current treatment such as occurred with oral laquinimod.


Compared to say interferons/glaterimer acetate that are low hisk-modest gain there are a number of high risk-high gain agents such as alemtuzumab/rituzimab (yet to be licensed) and tysabri it is perhaps easier to see why this trial design of high-risk drugs compared to low-risk drugs occurs, but once we have a low risk-high gain drug especially if it is oral then we may have a game changer. Then it will not only be hard to justify placebo control, but also not having to test drug on top of the low-risk, high efficacy established drug. if the two drugs are say immunosuppressive it may be difficult to show efficacy without increasing the risk of side-effects due to reduction in normal immune function. Therefore, once one of these drugs are shown to be effective and safe, it is going to be hard to knock the drug off its perch and develop new drugs Therefore, it is surprising that pharmaceutical companies are pricing their drugs so high, such that it limits uptake of the drug.


Therefore, there may be a limited window of opportunity to test nutriceuticals such as probiotics and vitamin D that will be of very low risk, but maybe of modest efficacy (based on animal studies) before a pharmaceutical becomes established, first-line treatments

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